Estimating the Costs of Cell and Gene Therapy Products: A Systematic Review

OBJECTIVES: Advances in cell and gene therapies offer the potential to cure different types of diseases and further improve health outcomes. However, access to these therapies is limited by their often-high costs. This review aims to compare methodologies used in estimating the costs of cell and gene therapy products.

METHODS: We conducted a systematic review of studies published in PubMed, EMBASE, and Web of Science that estimated the product costs of cell and gene therapies from inception to January 2024. Relevant publications were subjected to full text screening to assess eligibility and characteristics. We conducted hierarchical regression to analyze cost estimates while accounting for the potential effects of other variables.

RESULTS: A total of 5,890 publications were initially identified, of which 81 studies were found eligible, covering 129 cost estimates of cell and gene therapies. Cost estimation strategies included list prices for the actual or a similar product (n=69), cost estimation frameworks (n=37), and other estimation strategies (n=23). Median costs were EUR 295,000 and ranged from as low as EUR 1,500 for some types of cell-based therapy, to estimates by experts of as high as EUR 2,300,000 for gene therapy. Product costs estimated using list prices were about four times (p=0.006) higher than costs estimated using frameworks. Cost estimates from North America were about 60 percent higher than those from Europe (p=0.03). Estimated costs of gene therapies were about seven times higher (p≤0.001) than cell therapies and about three times higher (p≤0.001) than CAR T-Cell therapies.

CONCLUSIONS: The results of this review indicate a heterogeneity in methods and a wide range of cost estimates. The appropriate costing strategy varies with the stage of product development, with products in early development having more uncertainty compared to those already on the market.